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January 15, 2013

Genetic modification and stem cell treatment costs

Sangamo may have a "functional cure" for HIV. It is currently in two Phase 2 studies designed to maximize the engraftment of CCR5-disrupted T-cells. The two studies, which will focus on reduction or elimination of detectable virus in treated patients, are expected to have preliminary data in the first half of 2013 and final data later that year.

If successful in clinical trials the Sangamo treatment would be a once- or twice-a-year genetic-modification (modified T-cells) treatment. It might be more acceptable to patients than the current best treatment (a cocktail of drugs therapy, which can cost up to $40,000 per year and cause toxic side effects).

Sangamo places about 10 billion T-Cells into the patient. Both CCR5 genes are mutated in 5-10 percent of the cells.

New CRISPR genetic modification would be cheaper, faster and more effective than the zinc finger method.

Sangamo's gene-modifications are so precise and nontoxic that the company and researchers worldwide are working not only to optimize ZFN (zinc finger) therapies for HIV, but also for hemophilia, Huntington's disease and hemoglobinopathies such as sickle cell anemia and beta-thalassemia.



About 18000 people in the US have Hemophilia. Some current treatment can cost $300,000 per year.

The worldwide prevalence of Huntington's Disease is 5-10 cases per 100,000 persons but varies greatly geographically as a result of ethnicity, local migration and past immigration patterns. The average life expectancy after diagnosis is about 20 years.

Stem cell treatments with fewer stem cells (say 200 million) can cost about $500 per injection for other kinds of treatments. There are a lot of unproven and fraudulent stem cell treatments.

In 2011, Sigma was selling its ZFN gene modification kits for as high as $35,000 each to biotech companies and $25,000 each to academic institutions. Genetic modification kits (Zinc Finger or TALENs) for performing a specific gene editing now can cost about $3000-5000 each. The new CRISPR approach could reduce gene modification kit costs by 10 times.

Stem cell treatment could become lower cost and more standardized in the future with the isolation of more standard stem cell lines that can be used for patients. A japanese researcher is isolation about 75 stem cell lines that would be well tolerated by 80% of the population. Perhaps a couple hundred stem cell lines would be able to cover 95-98% of the population.



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